PH12018501711A1 - Compounds for treating spinal muscular atrophy - Google Patents

Compounds for treating spinal muscular atrophy

Info

Publication number
PH12018501711A1
PH12018501711A1 PH12018501711A PH12018501711A PH12018501711A1 PH 12018501711 A1 PH12018501711 A1 PH 12018501711A1 PH 12018501711 A PH12018501711 A PH 12018501711A PH 12018501711 A PH12018501711 A PH 12018501711A PH 12018501711 A1 PH12018501711 A1 PH 12018501711A1
Authority
PH
Philippines
Prior art keywords
compounds
muscular atrophy
spinal muscular
treating spinal
treating
Prior art date
Application number
PH12018501711A
Inventor
Hongyan Qi
Soongyu Choi
Amal Dakka
Gary Mitchell Karp
Jana Narasimhan
Nikolai Naryshkin
Anthony A Turpoff
Marla L Weetall
Ellen Welch
Matthew G Woll
Tianle Yang
Nanjing Zhang
Xiaoyan Zhang
Xin Zhao
Luke Green
Emmanuel Pinard
Hasane Ratni
Original Assignee
Hoffmann La Roche
Ptc Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Hoffmann La Roche, Ptc Therapeutics Inc filed Critical Hoffmann La Roche
Publication of PH12018501711A1 publication Critical patent/PH12018501711A1/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/70—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings condensed with carbocyclic rings or ring systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

Abstract

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
PH12018501711A 2012-02-10 2018-08-13 Compounds for treating spinal muscular atrophy PH12018501711A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261597523P 2012-02-10 2012-02-10
PCT/US2013/025292 WO2013119916A2 (en) 2012-02-10 2013-02-08 Compounds for treating spinal muscular atrophy

Publications (1)

Publication Number Publication Date
PH12018501711A1 true PH12018501711A1 (en) 2019-09-23

Family

ID=48948163

Family Applications (2)

Application Number Title Priority Date Filing Date
PH12014501786A PH12014501786B1 (en) 2012-02-10 2014-08-07 Compounds for treating spinal muscular atrophy
PH12018501711A PH12018501711A1 (en) 2012-02-10 2018-08-13 Compounds for treating spinal muscular atrophy

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PH12014501786A PH12014501786B1 (en) 2012-02-10 2014-08-07 Compounds for treating spinal muscular atrophy

Country Status (30)

Country Link
US (6) US9586955B2 (en)
EP (2) EP2812004B1 (en)
JP (2) JP6092897B2 (en)
KR (2) KR102341596B1 (en)
CN (2) CN108299314A (en)
AR (1) AR092794A1 (en)
AU (2) AU2013216870B2 (en)
BR (1) BR112014019750B1 (en)
CA (1) CA2863874C (en)
CL (1) CL2014002100A1 (en)
CO (1) CO7061082A2 (en)
CR (1) CR20140376A (en)
DK (1) DK2812004T3 (en)
EA (2) EA037123B1 (en)
EC (1) ECSP14017269A (en)
ES (1) ES2697174T3 (en)
HK (1) HK1202077A1 (en)
HU (1) HUE039779T2 (en)
IL (2) IL233959A (en)
MA (1) MA35920B1 (en)
MX (2) MX354074B (en)
NZ (1) NZ628186A (en)
PE (1) PE20142364A1 (en)
PH (2) PH12014501786B1 (en)
PL (1) PL2812004T3 (en)
SG (2) SG10201609188WA (en)
TR (1) TR201813877T4 (en)
TW (2) TWI585085B (en)
UA (1) UA116981C2 (en)
WO (1) WO2013119916A2 (en)

Families Citing this family (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10327940B2 (en) 2008-11-09 2019-06-25 3D Systems, Inc. Spiral brace
BR112014016287B1 (en) 2011-12-30 2022-08-09 F. Hoffmann - La Roche Ag COMPOUND AND PHARMACEUTICAL COMPOSITION
WO2013112788A1 (en) 2012-01-26 2013-08-01 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
TR201813877T4 (en) * 2012-02-10 2018-11-21 Hoffmann La Roche COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY.
MX352962B (en) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compounds for treating spinal muscular atrophy.
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
SG11201504933WA (en) * 2012-12-24 2015-07-30 Univ Ramot Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
CN105392790B (en) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 The preparation of 4H- pyrido [1,2-a] pyrimidin-4-one compounds is used to prevent or the purposes of the drug for the treatment of cancer
US10195202B2 (en) 2013-12-19 2019-02-05 Ptc Therapeutics, Inc. Methods for modulating the amount of RNA transcripts
HUE046491T2 (en) * 2014-05-15 2020-03-30 Hoffmann La Roche Compounds for treating spinal muscular atrophy
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
KR102620328B1 (en) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 Targeted augmentation of nuclear gene output
WO2016111896A1 (en) * 2015-01-05 2016-07-14 Sikorsky Aircraft Corporation Integrated vibration damper for additively manufactured structure and method
WO2016184832A1 (en) 2015-05-20 2016-11-24 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017080967A1 (en) * 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
CN108137601A (en) * 2015-11-12 2018-06-08 豪夫迈·罗氏有限公司 For treating the compound of amyotrophic lateral sclerosis
CN113750101A (en) 2015-12-10 2021-12-07 Ptc医疗公司 Methods for treating huntington's disease
JP6872550B2 (en) 2015-12-10 2021-05-19 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ Crosslinked piperidine derivative
CN109312343B (en) 2015-12-14 2022-09-27 冷泉港实验室 Antisense oligomers for the treatment of autosomal dominant mental retardation type 5 and Dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017186668A1 (en) 2016-04-28 2017-11-02 F. Hoffmann-La Roche Ag A process for the preparation of 2-pyrazolo[1,5-a]pyrazin-2-ylpyrido[1,2-a]pyrimidin-4-one
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
CA3043755A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
US11274107B2 (en) 2016-12-22 2022-03-15 Cadent Therapeutics, Inc. NMDA receptor modulators and uses thereof
SG11201911615WA (en) 2017-06-05 2020-01-30 Ptc Therapeutics Inc Compounds for treating huntington's disease
EP3638318A4 (en) 2017-06-14 2021-03-17 PTC Therapeutics, Inc. Methods for modifying rna splicing
CA3067592A1 (en) * 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
MX2019015578A (en) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Methods for treating huntington's disease.
SG10202002990XA (en) 2017-08-04 2020-05-28 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
SG10202108375XA (en) 2017-08-25 2021-09-29 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
BR112020005549A2 (en) * 2017-09-22 2020-10-06 F. Hoffmann-La Roche Ag process for the preparation of 7- (4,7-diazaspiro [2.5] octan-7-yl) -2- (2,8-dimethylimidazo [1,2-b] pyridazin-6-yl) pyrido derivatives [1, 2-a] pyrimidin-4-one
MX2020009957A (en) 2018-03-27 2021-01-15 Ptc Therapeutics Inc Compounds for treating huntington's disease.
MX2020014116A (en) * 2018-06-27 2021-06-15 Reborna Biosciences Inc Prophylactic or therapeutic agent for spinal muscular atrophy.
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
PE20211378A1 (en) 2018-06-27 2021-07-27 Ptc Therapeutics Inc HETEROCYCLIC AND HETEROARYL COMPOUNDS TO TREAT HUNTINGTON DISEASE
DK3830092T3 (en) 2018-08-03 2023-08-07 Novartis Ag Heteroaromatic nmda receptor modulators and uses thereof
WO2020033473A1 (en) * 2018-08-07 2020-02-13 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
JP2022521467A (en) 2019-02-05 2022-04-08 スカイホーク・セラピューティクス・インコーポレーテッド Methods and compositions for regulating splicing
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN110540535B (en) * 2019-10-23 2020-07-31 上海再启生物技术有限公司 Process suitable for preparing 4- (6-aminopyridin-3-yl) substituted piperidine in amplification way
CN111116576A (en) * 2019-12-01 2020-05-08 北京师范大学 Quinolizinone compound and preparation method thereof
WO2021111124A1 (en) 2019-12-02 2021-06-10 Storm Therapeutics Limited Polyheterocyclic compounds as mettl3 inhibitors
EP4097092A1 (en) 2020-01-28 2022-12-07 Protego Biopharma, Inc. Compounds, compositions and methods for stabilizing transthyretin and inhibiting transthyretin misfolding
US20230365566A1 (en) 2020-02-28 2023-11-16 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
CR20220485A (en) 2020-02-28 2022-11-10 Ionis Pharmaceuticals Inc Compounds and methods for modulating smn2
US20230365526A1 (en) 2020-02-28 2023-11-16 Remix Therapeutics Inc. Pyridazine derivatives for modulating nucleic acid splicing
AU2021228285A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
KR20220167298A (en) 2020-04-08 2022-12-20 레믹스 테라퓨틱스 인크. Compounds and methods for modulating splicing
CN116134036A (en) 2020-04-08 2023-05-16 雷密克斯医疗公司 Compounds and methods for modulating splicing
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc Opa1 antisense oligomers for treatment of conditions and diseases
WO2022006550A1 (en) 2020-07-02 2022-01-06 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
JP2023532331A (en) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modifiers for splicing nucleic acids and for treating proliferative diseases and compounds and methods for modulating splicing
WO2022070071A1 (en) * 2020-09-30 2022-04-07 Janssen Biotech, Inc. Dihydroorotate dehydrogenase inhibitors
JP2024514421A (en) 2021-03-17 2024-04-02 エフ． ホフマン－ラ ロシュ アーゲー New thiazolopyrimidinone derivatives
WO2023009585A2 (en) 2021-07-28 2023-02-02 Protego Biopharma, Inc. Transthyretin stabilizing compounds
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034833A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034811A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034812A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3233973A1 (en) * 2021-10-13 2023-04-20 Dominic Reynolds Compounds and methods for modulating nucleic acid splicing
TW202330552A (en) 2021-10-13 2023-08-01 美商雷密克斯醫療公司 Compounds and methods for modulating splicing
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202346305A (en) 2022-01-05 2023-12-01 美商雷密克斯醫療公司 Compounds and methods for modulating splicing
WO2023133217A1 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
CN117263936B (en) * 2023-11-21 2024-02-23 中国中医科学院医学实验中心 Imidazo [1,2-a ] pyridine derivative, preparation method thereof and application thereof in drug for inhibiting central nervous system penetrating HDAC6

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3558618A (en) * 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
US4122274A (en) * 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
US4342870A (en) * 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
JPS56150091A (en) * 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5726182A (en) * 1990-05-02 1998-03-10 Abbott Laboratories Quinolizinone type compounds
EP0640083A1 (en) * 1992-05-13 1995-03-01 E.I. Du Pont De Nemours And Company Substituted pyrido 1,2-a]pyrimidinone derivatives as fungicides
JPH11510478A (en) * 1995-06-06 1999-09-14 アボツト・ラボラトリーズ Quinolidinone type compounds
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
WO2000017197A1 (en) * 1998-09-21 2000-03-30 Biochem Pharma Inc. Quinolizinones as integrin inhibitors
WO2001030757A1 (en) * 1999-10-28 2001-05-03 Microcide Pharmaceuticals, Inc. Drug discharge pump inhibitors
AU2001230426C1 (en) * 2000-01-24 2006-06-22 Astrazeneca Ab Therapeutic morpholino-substituted compounds
WO2002087589A1 (en) * 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Medicine for inhibiting drug elimination pump
CA2469316A1 (en) 2001-12-07 2003-06-19 Vertex Pharmaceuticals, Inc. Pyrimidine-based compounds useful as gsk-3 inhibitors
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
WO2004009558A2 (en) 2002-07-24 2004-01-29 Ptc Therapeutics, Inc. Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations
CN1809563A (en) * 2003-06-20 2006-07-26 希龙公司 Pyridino[1,2-a]pyrimidin-4-one compounds as anticancer agents
CA2563669A1 (en) * 2004-05-04 2005-11-10 Warner-Lambert Company Llc Pyrrolyl substituted pyrido[2,3-d]pyrimidin-7-ones and derivatives thereof as therapeutic agents
CN101146798A (en) 2005-01-21 2008-03-19 詹森药业有限公司 Novel heterocyclic benzo[C]chromene derivatives useful as modulators of the estrogen receptors
AR059339A1 (en) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd CUMARINE DERIVATIVES FOR PROLIFERATIVE DISORDERS OF CELLS, PHARMACEUTICAL COMPOSITION AND THERAPEUTIC AGENT CONTAINING THEM
US8110681B2 (en) * 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
WO2010019243A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
WO2011050245A1 (en) * 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
EP2501231B1 (en) 2009-11-20 2016-12-21 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
EP2523956A1 (en) * 2010-01-13 2012-11-21 Institut Pasteur Korea Anti - infective pyrido (1,2 -a) pyrimidines
BR112014016287B1 (en) 2011-12-30 2022-08-09 F. Hoffmann - La Roche Ag COMPOUND AND PHARMACEUTICAL COMPOSITION
WO2013112788A1 (en) 2012-01-26 2013-08-01 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
TR201813877T4 (en) * 2012-02-10 2018-11-21 Hoffmann La Roche COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY.
MX352962B (en) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compounds for treating spinal muscular atrophy.
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
HUE046491T2 (en) 2014-05-15 2020-03-30 Hoffmann La Roche Compounds for treating spinal muscular atrophy
WO2016184832A1 (en) 2015-05-20 2016-11-24 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy

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CN104349777A (en) 2015-02-11
PH12014501786A1 (en) 2014-11-10
BR112014019750A2 (en) 2017-06-20
KR102341596B1 (en) 2021-12-21
IL254045B (en) 2019-05-30
PH12014501786B1 (en) 2014-11-10
TWI585085B (en) 2017-06-01
US11753407B2 (en) 2023-09-12
BR112014019750B1 (en) 2020-03-03
UA116981C2 (en) 2018-06-11
CN108299314A (en) 2018-07-20
CR20140376A (en) 2015-01-23
IL233959A (en) 2017-11-30
AU2013216870B2 (en) 2017-07-20
US20180105526A1 (en) 2018-04-19
NZ628186A (en) 2016-03-31
HK1202077A1 (en) 2015-09-18
KR20140121482A (en) 2014-10-15
KR102137087B1 (en) 2020-07-24
EA201792465A1 (en) 2018-08-31
CA2863874A1 (en) 2013-08-15
IL233959A0 (en) 2014-09-30
WO2013119916A2 (en) 2013-08-15
TR201813877T4 (en) 2018-11-21
CN104349777B (en) 2018-05-01
PL2812004T3 (en) 2019-01-31
KR20200093066A (en) 2020-08-04
US9879007B2 (en) 2018-01-30
EP3406252A1 (en) 2018-11-28
EA201491505A1 (en) 2015-01-30
TW201336842A (en) 2013-09-16
DK2812004T3 (en) 2018-10-15
JP6363744B2 (en) 2018-07-25
CL2014002100A1 (en) 2015-10-23
AU2017204248B2 (en) 2019-05-30
EP3406252B1 (en) 2020-05-13
WO2013119916A3 (en) 2013-10-24
CA2863874C (en) 2021-02-16
JP2017122097A (en) 2017-07-13
US20240067646A1 (en) 2024-02-29
JP6092897B2 (en) 2017-03-08
AR092794A1 (en) 2015-05-06
EP2812004A2 (en) 2014-12-17
MA35920B1 (en) 2014-12-01
EA029542B1 (en) 2018-04-30
MX354074B (en) 2018-02-12
TWI629274B (en) 2018-07-11
HUE039779T2 (en) 2019-02-28
EP2812004A4 (en) 2015-11-11
US20190375750A1 (en) 2019-12-12
JP2015508075A (en) 2015-03-16
PE20142364A1 (en) 2015-01-10
BR112014019750A8 (en) 2018-01-16
IL254045A0 (en) 2017-10-31
ES2697174T3 (en) 2019-01-22
TW201722942A (en) 2017-07-01
ECSP14017269A (en) 2015-09-30
US20170129885A1 (en) 2017-05-11
AU2013216870A1 (en) 2014-08-28
US10851101B2 (en) 2020-12-01
EP2812004B1 (en) 2018-06-27
AU2017204248A1 (en) 2017-07-13
US20210276999A1 (en) 2021-09-09
MX2022011699A (en) 2022-11-09
EA037123B1 (en) 2021-02-09
SG10201609188WA (en) 2016-12-29
CO7061082A2 (en) 2014-09-19
MX2014009696A (en) 2015-01-14
US9586955B2 (en) 2017-03-07
US20150005289A1 (en) 2015-01-01
SG11201404713PA (en) 2014-09-26

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